Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort
| العنوان: | Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort |
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| المؤلفون: | Rita Beier, Marina Cavazzana, Figen Dogu, Yves Bertrand, Paul Veys, Francesca Ferrua, Robbert G. M. Bredius, Roland Meisel, Arnalda Lanfranchi, Renata Formankova, Stéphane Blanche, Virginie Courteille, Elena Soncini, Tayfun Güngör, Jolanta Gozdzik, Kim Vettenranta, Krzysztof Kałwak, Mikael Alligon, Natacha Entz-Werle, Ansgar Schulz, Nizar Mahlaoui, Savaş Kansoy, Wilhelm Friedrich, Amos Toren, Mehmet A. Yeşilipek, Alina Ferster, Andrew R. Gennery, Mary Slatter, Despina Moshous, Fulvio Porta, Marco Zecca, Anders Fasth, Karoline Ehlert, Gérard Michel, Bénédicte Neven, Victoria Bordon, Alphan Kupesiz, Mikael Sundin, Kanchan Rao, Cristina Diaz-de-Heredia, Isabelle Badell Serra, Michael H. Albert, Herbert Pichler, Arjan C. Lankester, Andrew J. Cant, Marta González-Vicent, Petr Sedlacek, Jose Moraleda, Caroline A. Lindemans, Peter Bader, Manfred Hoenig, Alain Fischer, Austen Worth, Dmitry Balashov, Erik G J von Asmuth, Carsten Speckmann, Nuno Miranda, Aydan Ikinciogullari |
| المساهمون: | Clinicum, Children's Hospital, Lastentautien yksikkö, HUS Children and Adolescents, University of Zurich, Lankester, Arjan C, Institut Català de la Salut, [Lankester AC, von Asmuth EGJ] Pediatric Stem Cell Transplantation Program and Laboratory for Pediatric Immunology, Willem-Alexander Children’s Hospital, Leiden University Medical Center, Leiden, The Netherlands. [Neven B] Unité d’Immuno-hematologie et Rhumatologie Pédiatrique, Hôpital Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France. Université de Paris, Paris, France. Institut Imagine, INSERM UMR1163, Laboratory of Immunogenetics of Pediatric Autoimmune Diseases, Paris, France. [Mahlaoui N, Courteille V, Alligon M] French National Reference Center for Primary Immunodeficiencies (CEREDIH) and European Registry for Stem Cell Transplantation for Primary Immunodeficiencies (SCETIDE), Hôpital Universitaire Necker-Enfants malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France. [Diaz-de-Heredia C] Servei d’Oncologia i Hematologia Pediàtriques, Vall d’Hebron Hospital Universitari, Barcelona, Spain, Vall d'Hebron Barcelona Hospital Campus |
| المصدر: | JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY r-IIB SANT PAU. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica Sant Pau Instituto de Salud Carlos III (ISCIII) Journal of Allergy and Clinical Immunology, 149(5), 1744-1754.e8. MOSBY-ELSEVIER Scientia |
| بيانات النشر: | Elsevier BV, 2022. |
| سنة النشر: | 2022 |
| مصطلحات موضوعية: | Oncology, Transplantation Conditioning, medicine.medical_treatment, Hematopoietic stem cell transplantation, Cohort Studies, 0302 clinical medicine, conditioning, Immunology and Allergy, OUTCOMES, 0303 health sciences, Otros calificadores::Otros calificadores::/genética [Otros calificadores], Hematopoietic Stem Cell Transplantation, immune reconstitution, 3. Good health, surgical procedures, operative, medicine.anatomical_structure, Cohort, 2723 Immunology and Allergy, SURVIVAL, Malalties congènites, Unrelated Donors, medicine.medical_specialty, Immunology, 610 Medicine & health, pretransplantation infections, SCID, 03 medical and health sciences, Internal medicine, enfermedades y anomalías neonatales congénitas y hereditarias::enfermedades del recién nacido::inmunodeficiencia combinada grave [ENFERMEDADES], Other subheadings::Other subheadings::/genetics [Other subheadings], medicine, Humans, genetic subgroups, Interleukin-7 receptor, 030304 developmental biology, Congenital, Hereditary, and Neonatal Diseases and Abnormalities::Infant, Newborn, Diseases::Severe Combined Immunodeficiency [DISEASES], 2403 Immunology, Severe combined immunodeficiency, Cèl·lules mare hematopoètiques - Trasplantació, business.industry, medicine.disease, Anti-thymocyte globulin, Transplantation, RECONSTITUTION, Graft-versus-host disease, 10036 Medical Clinic, 3121 General medicine, internal medicine and other clinical medicine, Severe Combined Immunodeficiency, Bone marrow, business, 030215 immunology |
| الوصف: | Genetic subgroups; Immune reconstitution; Pretransplantation infections Subgrupos genéticos; Reconstitución inmune; Infecciones previas al trasplante Subgrups genètics; Reconstitució immune; Infeccions prèvies al trasplantament Background Hematopoietic stem cell transplantation (HSCT) represents a curative treatment for patients with severe combined immunodeficiency (SCID), a group of monogenic immune disorders with an otherwise fatal outcome. Objective We performed a comprehensive multicenter analysis of genotype-specific HSCT outcome, including detailed analysis of immune reconstitution (IR) and the predictive value for clinical outcome. Methods HSCT outcome was studied in 338 patients with genetically confirmed SCID who underwent transplantation in 2006-2014 and who were registered in the SCETIDE registry. In a representative subgroup of 152 patients, data on IR and long-term clinical outcome were analyzed. Results Two-year OS was similar with matched family and unrelated donors and better than mismatched donor HSCT (P < .001). The 2-year event-free survival (EFS) was similar in matched and mismatched unrelated donor and less favorable in mismatched related donor (MMRD) HSCT (P < .001). Genetic subgroups did not differ in 2-year OS (P = .1) and EFS (P = .073). In multivariate analysis, pretransplantation infections and use of MMRDs were associated with less favorable OS and EFS. With a median follow-up of 6.2 years (range, 2.0-11.8 years), 73 of 152 patients in the IR cohort were alive and well without Ig dependency. IL-2 receptor gamma chain/Janus kinase 3/IL-7 receptor–deficient SCID, myeloablative conditioning, matched donor HSCT, and naive CD4 T lymphocytes >0.5 × 10e3/μL at +1 year were identified as independent predictors of favorable clinical and immunologic outcome. Conclusion Recent advances in HSCT in SCID patients have resulted in improved OS and EFS in all genotypes and donor types. To achieve a favorable long-term outcome, treatment strategies should aim for optimal naive CD4 T lymphocyte regeneration. |
| وصف الملف: | PIIS0091674921016298.pdf - application/pdf; application/pdf |
| تدمد: | 0091-6749 |
| الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c3a166208b4a369dac2c837c1851bc17Test https://doi.org/10.1016/j.jaci.2021.10.017Test |
| حقوق: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....c3a166208b4a369dac2c837c1851bc17 |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 00916749 |
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